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Notification report


Full notification file


General information

Notification Number
B/ES/17/16

Member State to which the notification was sent
Spain

Date of acknowledgement from the Member State Competent Authority
04/09/2017

Title of the Project
A Global, Open-Label, Multicenter, Phase 1/2 Study of the Safety and Dose Escalation of BAX 888, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing В-Domain Deleted Factor VIII (BDD-FVIII) in Severe Hemophilia A Subjects Administered a Single Intravenous Infusion

Proposed period of release:
30/12/2017 to 30/06/2022

Name of the Institute(s) or Company(ies)
Baxalta Innovations GmbH, ;


3. Is the same GMO release planned elsewhere in the Community?
Yes:
Austria; Germany; France; Italy; Hungary;

Has the same GMO been notified elsewhere by the same notifier?
Yes

If yes, notification number(s):


GMO characterization

GMO is a:
DNA Virus

Identity of the GMO:
BAX 888 is an AAV vector containing single-stranded DNA carrying the B-domain deleted (BDD) human coagulation F8 (BDD-FVIII) cDNA. BAX 888 has an INFORMATION DELETED FOR CONFIDENTIALITY PURPOSES AAV capsid composed of proteins belonging to the AAV serotype 8 (AAV8).

Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
AAV 8DependoparvovirusAdeno-associated Virus---

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known